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Angelman Syndrome Relevant Outcome Measures in a Novel Rat Model

$148,810 2017 FT2017-002: 2017-2018

Angelman Syndrome Relevant Outcome Measures in a Novel Rat Model

Novel strategies to treat Angelman syndrome are needed as there is currently no transformative corrective therapy, and supportive care only marginally alleviates symptoms. If normal levels of ubiquitin ligase protein E3A (UBE3A) are expressed, regression of pervasive disease pathology and behavioral symptoms is an achievable goal. Advances in rat genetic models, facilitated by FAST, herald a new era of behavioral genetics. Concomitantly, we seized the opportunity, by developing sophisticated assays for the more appropriate model species, to facilitate the discovery of treatments for disorders with social communication and intellectual disability. The goal is to utilize UC Davis’ innovative outcome measures to identify AS relevant functional phenotypes in the Ube3a mutant rat. Future directions will test a variety of therapeutics, by measuring acoustic social communication and touchscreen based learning and memory.

Principle Investigator

Jill Silverman, PhD

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